R.M. Preshaw: The multiple sclerosis liberation therapy fiasco

The multiple sclerosis liberation therapy fiasco

Clinical trials of treating multiple sclerosis (MS) by dilating neck veins with balloons (so-called liberation therapy) have been unsuccessful. Results from an outstanding, but small, trial in Buffalo, New York showed no value to the treatment. This mirrored the findings of other experts.

For several years an Italian vascular surgeon, Paolo Zamboni, promoted a theory that MS is due to constriction in veins leading from the brain. In 2009, he published anecdotal results from a small number of patients in an obscure journal in which he claimed improvements when these veins were dilated, and in some cases, kept open with surgical stents. It is estimated that over 20,000 patients with MS have now undergone this procedure in more than 30 countries, including a few here in Canada. Other Canadians received the treatment abroad.

Canada has one of the world’s highest rates of MS, and interest in this treatment was hyped by enthusiastic media coverage. Pressure groups (mainly patients and their relatives) persuaded both provincial and federal politicians to expend tax dollars in pursuit of results favouring this treatment — despite almost uniform reservations expressed by physicians with expertise in treatment of this baffling disease.

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There will still be many who will want to believe in the efficacy of this treatment, but the facts are in. Liberation therapy doesn’t work. It is now time to consider what we can learn from this sad fiasco.

The combination of clinical research agencies and the pharmaceutical industry appears able to determine worldwide, with fair reliability, the value (or otherwise) of new drugs. This success is due to the development over the last century of sophisticated clinical trials which compare new drugs with placebos (or older drugs). Confirmation of results by further trials, often in other countries, is an integral part of this process.

Yet trials of interventional treatments, such as surgery, cannot be conducted in the same way. There is usually no such thing as a placebo surgery. In liberation therapy, however, it was possible to perform a sham operation — that is, sedate the subject and pass guide wires into the veins leading from the brain, but omit the crucial step of dilating these veins with a balloon. Not surprisingly, there were few volunteers. As an MS patient, would you volunteer when the sham procedure has risks of its own (including death), with no prospect of any benefit?

Intriguingly, countries with no national health system dealt with the high hopes for liberation therapy without much difficulty or disruption. In the United States, there was no allocation of funds for this treatment by state and federal authorities (although the MS Society committed a large fraction of its budget to such research). The US Food and Drug Agency simply issued a statement in May 2012 advising that the evidence in favour of liberation therapy did not exist, and the risks of the treatment included serious injuries and death. Some Americans (and no doubt some Canadians) did pay for the treatment in American hospitals, out of pocket. But with no government funding available, and insurance companies reluctant to fund unproven treatments, liberation therapy did not take off in the U.S.

In Canada, and other countries with public healthcare systems, however, MS patients demanded — often successfully — that governments provide funds for these experimental procedures. Politicians proved a softer touch than insurance companies.

But in publicly funded systems, like Canada’s, every dollar spent on liberation therapy was a dollar taken from something else. The liberation treatment fiasco demands that healthcare expenditures be determined by experts, bound by rules of evidence. This is especially important considering the tremendous strain that healthcare expenses are placing on provincial treasuries all across the country. There isn’t enough money to fully fund all the medical treatments we know work. We literally cannot afford to spend precious resources on therapies that have yet to be proven effective.

The need to properly vet new and experimental procedures suggests a way in which our healthcare system can be streamlined and improved. Currently, each province and territory approves new drugs independently, after preliminary licencing by Health Canada (this sometimes results in sad stories of desperately ill Canadians being denied badly needed medicines that are available in Alberta, for instance, but not yet approved in their own home province). The time has come to centralize the approval of new drugs and therapies at the federal level.

In Britain, the National Health Service has already established a committee, the National Institute for Health and Care Excellence (NICE), which makes recommendations — sometimes contentious — on which health interventions to support. Although the decisions of NICE are clearly not applicable to other jurisdictions, it still provides a model for sorting through the high volume of new drugs and treatments in an organized, evidence-based manner. (Incidentally, a year ago, NICE stated that liberation therapy for MS should only be performed as part of a clinical trial — but that such trials themselves were not recommended).

Indeed, there is room for co-operation here not just on the national level, but also the international. Advanced countries could easily pool resources and findings, quickly determining which new treatments are worthy and which are ineffective or, worse, harmful. The Cochrane Collaboration is such an international group, which, despite limited financial resources, manages to provide reliable assessments of many areas of health care. (This group, too, also found no benefit to liberation therapy.)

The sad truth is that there never was any convincing evidence for the relatively large expense of clinical trials of this treatment

Such attempts at assessment should not be restricted to drugs. They should also consider interventional treatments, both new and old. Dr. Robert McMurtry, past president of the Canadian Orthopaedic Association, has publicly stated that perhaps as many as 30% of elective surgical procedures may be providing no real benefit. For example, arthroscopic surgery on degenerative joint disease in the knee offers no improvement over a conservative approach, but such procedures continue to be common. According to a 2013 study from Edmonton, use of expensive magnetic resonance imaging (MRI) scans for investigation of chronic back pain was inappropriate in over half the patients reviewed. In 2008, the Vancouver Island Health Authority funded hundreds of patients’ virtual colonoscopies by means of CT scans, a screening procedure with no established benefit either then or since.

Some will object to such overweening government interference in health care, and point out that if a new and novel treatment is found, some patients may die before the treatment meets the approval of a central agency. That is indeed certainly possible. But that sad reality must be weighed against the costs, in both money and potentially lives, of providing drugs and treatments that are ineffective or outright harmful.

Some authorities, even reputable ones, continue to recommend that liberation therapy be further studied. This is understandably leaped upon by MS victims and their loved ones, who continue to hope for a breakthrough treatment for this cruel disease.

But the sad truth is that there never was any convincing evidence for the relatively large expense of clinical trials of this treatment, or for raising the hopes for those touched by MS. Liberation therapy is not an effective treatment for this disease. The fight for a cure will continue, but along other avenues.

National Post

R.M. Preshaw is a Canadian doctor, who serves on a B.C. Medical Association Committee examining allied health services.

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